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Genome editing, or genome engineering, or gene editing, is a type of genetic engineering in which DNA is inserted, deleted, modified or replaced in the genome of a living organism. Unlike early genetic engineering techniques that randomly inserts genetic material into a host genome, genome editing targets the insertions to site-specific locations. The basic mechanism involved in genetic manipulations through programmable nucleases is the recognition of target genomic loci and binding of effector DNA-binding domain (DBD), double-strand breaks (DSBs) in target DNA by the restriction endonucleases, and the repair of DSBs through homology-directed recombination (HDR) or non-homologous end joining (NHEJ).
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CRISPR Therapeutics AG is a Swiss–American biotechnology company headquartered in Zug, Switzerland. It was one of the first companies formed to utilize the CRISPR gene editing platform to develop medicines for the treatment of various rare and common diseases. The company has approximately 500 employees and has offices in Zug, Switzerland, Boston, Massachusetts, San Francisco, California and London, United Kingdom. Its manufacturing facility in Framingham, Massachusetts won the Facilities of the Year Award (FOYA) award in 2022. The company’s lead program, Exagamglogene autotemcel, or exa-cel, has been submitted for regulatory approval in 2023.
Maria Grazia Roncarolo is an Italian pediatrician who is currently George D. Smith Professor in Stem Cell and Regenerative Medicine and Professor of Medicine at Stanford University. She is also the Director of the Stanford Institute of Stem Cell Biology and Regenerative Medicine along with Irving Weissman and Michael Longaker and the Director for Center for Definitive and Curative Medicine at Stanford.
Precision BioSciences, Inc. is a publicly traded American clinical stage gene editing company headquartered in Durham, North Carolina. Founded in 2006, Precision is focused on developing both in vivo and ex vivo gene editing therapies using its proprietary "ARCUS" genome editing platform.
References
- ↑ "Matthew Porteus". Stanford University School of Medicine . Retrieved 23 June 2021.
- ↑ Porteus MH, Baltimore D (May 2003). "Chimeric nucleases stimulate gene targeting in human cells". Science. 300 (5620): 763. doi:10.1126/science.1078395. PMID 12730593. S2CID 34460337.
- 1 2 "Dr. Matthew Porteus". CRISPR Therapeutics . Retrieved 23 June 2021.
- ↑ "There Are Now Two Pushes to Treat Sickle Cell Disease With CRISPR". CRISPR Medicine News. Retrieved 26 June 2021.
- ↑ "About Us: Academic Founders". Graphite Bio. Retrieved 26 June 2021.
- ↑ "Matthew Porteus". Google Scholar . Retrieved 28 October 2023.
